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The Hospital for Sick Children


Global trial supported by SickKids stem cell research shows targeted heart drug reduces obstructive HCM in youth

A global clinical trial led by SickKids has found that mavacamten improves blood flow and reduces obstruction in youth with hypertrophic cardiomyopathy (HCM), a genetic heart disease. The study's results offer hope to families avoiding open-heart surgery, setting the stage for precision therapeutics.

SourceThe Hospital for Sick Children·JournalNew England Journal of Medicine·TypeRandomized controlled/clinical trial·DateMar 29, 2026

Novel genes linked to rare childhood diarrhea

Researchers at SickKids identified three new genes associated with rare childhood diarrhea, providing a diagnosis for 48% of cases. The study found that genetic testing and targeted treatments can improve the quality of life for children like Sophie, who was diagnosed with CODE at just two days old.

SourceThe Hospital for Sick Children·JournalNew England Journal of Medicine·DateApr 2, 2025

Genetic discovery links new gene to autism spectrum disorder

A new genetic link has been identified between the DDX53 gene and autism spectrum disorder (ASD), providing crucial insights into the biological underpinnings of the condition. The study found that variants in the DDX53 gene contribute to ASD, particularly in males, highlighting its potential role in the male predominance observed in ASD.

SourceThe Hospital for Sick Children·JournalAmerican Journal of Human Genetics·DateDec 19, 2024

Global clinical trial shows improved survival rates for common childhood leukemia

A global clinical trial has shown a significant improvement in disease-free survival rates for children with B-cell acute lymphoblastic leukemia (B-ALL) by combining standard chemotherapy with blinatumomab, an immunotherapy. The study found improved survival rates of up to 97.5% after three years, compared to 90% with chemotherapy alone.

SourceThe Hospital for Sick Children·JournalNew England Journal of Medicine·DateDec 7, 2024

SickKids program provides integrated, trauma-informed care for pregnant and parenting adolescents

A recent study from The Hospital for Sick Children found that pregnant teenagers in Ontario were at a 50% higher risk of premature death before age 31 compared to those with no teen pregnancy. The Young Prenatal Program and Young Families Program offer integrated physical, mental, and developmental care to support adolescent parents.

SourceThe Hospital for Sick Children·JournalJAMA Network Open·DateMar 14, 2024

Overcoming barriers to conducting clinical trials in childhood rare disease research

A team of scientists at The Hospital for Sick Children developed an innovative statistical approach to demonstrate the effectiveness of a medication in reducing disease progression and liver transplants in children with Alagille syndrome. Using data from an international real-world cohort, they found a 70% improvement in event-free sur...

SourceThe Hospital for Sick Children·JournalHepatology·DateFeb 29, 2024